Note: This story was updated Oct. 22, 2024, to remove a reference to the therapy being pulled from the market in Europe. Although this was the initial recommendation, the therapy has remained available in Europe.  PTC Therapeutics plans to soon ask the U.S. Food and Drug Administration (FDA)…

My family has had a hectic week. It started out lovely; the entire family, all nine of us, spent a beautiful, springlike day together at the zoo. By Sunday evening, however, we had kids with fevers, coughs, and runny noses. At last count, four of us have come down with…

The adjective “rare” refers to an event, situation, or condition not occurring very often, not found in large numbers, and consequently of interest. For example, Duchenne muscular dystrophy (DMD) is a rare disease affecting approximately 1 in 3,500 male births worldwide. Years ago, on one of the first…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label update for Translarna (ataluren) to enable its use in Duchenne muscular dystrophy (DMD) patients who lost their ability to walk. The committee has specifically voted to remove…

Translarna (ataluren) significantly improves physical fitness in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), a study examining multiple endpoints across two trials shows. The study used a statistical strategy for analyzing multiple trial endpoints together, a tactic that could improve the interpretation…

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended expanding the use of Translarna (ataluren) to ambulatory children ages 2-5 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD). CHMP’s recommendation of Translarna use in younger children is based…

PTC Therapeutics is recruiting Duchenne muscular dystrophy (DMD) patients ages 5 and older and with a nonsense mutation for a long-term Phase 3 study to characterize the effects of Translarna (ataluren) on disease progression. The trial (NCT03179631) opened in July 2017 and enrolling patients at eight sites across…

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory…

A U.S. Food and Drug Administration (FDA) advisory board concluded that evidence supporting the effectiveness of Translarna (ataluren), by PTC Therapeutics, as a treatment for Duchenne muscular dystrophy is inconclusive. The opinion, which was endorsed by 10 of the 11 advisory board members at its Sept. 28 meeting, also stated that…

PTC Therapeutics shared additional data from a Phase 3 trial of Translarna (ataluren), showing that the treatment slightly slowed disease progression in ambulatory boys with Duchenne muscular dystrophy. The data was presented at the recent American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting in Phoenix, Arizona.